The US Food and Drug Administration (FDA) has granted breakthrough therapy designation for British biopharmaceutical firm Adaptimmune Therapeutics’ affinity enhanced T-cell therapy targeting NY-ESO in synovial sarcoma.
The therapy is designed for HLA-A*201, HLA-A*205 or HLA-A*206 allele-positive patients with inoperable or metastatic synovial sarcoma who have received prior chemotherapy and whose tumour expresses the NY-ESO-1 tumour antigen.
The breakthrough status was based on the results of a Phase I / II trial in patients with unresectable, metastatic or recurrent synovial sarcoma who have received prior chemotherapy.
In the trial, patients were treated with lymphodepleting chemotherapy followed by immunotherapy with T-cells engineered to recognise an HLA-A2 restricted NY-ESO-1 peptide.
Adaptimmune chief medical officer Dr Rafael Amado said: "We are committed to investigating the potential of our NY-ESO-1-T cell therapy across a variety of cancers.
"We are pleased that the FDA has granted Breakthrough Therapy designation for our T-cell therapy in synovial sarcoma, recognising both the unmet need for patients suffering from this disease as well as the promise of these early data.
"We look forward to working closely with the FDA to expedite the clinical development of this therapeutic candidate."
The company reported that in the primary efficacy analysis, 60% of the ten patients receiving the target dose of cells responded, and there was a 50% overall response rate in the 12 patients receiving any dose of cells.
The most common adverse events observed in the Phase I / II trial included nausea, anemia, pyrexia, lymphopenia and neutropenia.
It is reported that cytokine release syndrome (CRS) was seen in four of twelve subjects, with grade three CRS observed in two subjects, however no grade four CRS events were observed.
Recently, the company has announced its plan to begin pivotal studies with its affinity enhanced T-cell therapy targeting NY-ESO in synovial sarcoma by the end of this year, and that it will also explore development in myxoid round cell liposarcoma.
Studies with this therapy are also being carried out in myeloma, melanoma, ovarian cancer and non-small cell lung cancer.
Image: Micrograph of a monophasic synovial sarcoma. Photo: courtesy of Nephron.
